SAN DIEGO — October 5, 2026
Capricor Therapeutics announced positive new data from the HOPE-3 open-label extension (OLE) study evaluating Deramiocel in patients with Duchenne muscular dystrophy (DMD). The 24-month crossover analysis showed that patients who switched from placebo to Deramiocel after one year experienced a 76% reduction in the rate of upper limb functional decline compared with their own decline during the preceding placebo year. At Month 24, both treatment groups also showed slower upper limb decline than predicted by natural-history models. The findings were presented as a late-breaking poster at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan.
Deramiocel Slows Upper Limb Functional Decline
The HOPE-3 OLE analysis evaluated upper limb function using the Performance of the Upper Limb (PUL 2.0) total score. Patients originally assigned to placebo experienced a least-squares mean decline of 2.05 points during Year 1 on placebo, followed by a 0.49-point decline during Year 2 after switching to Deramiocel, representing a 76% reduction in the rate of decline. Patients who received Deramiocel from the beginning showed similar rates of decline across both years, with changes of 0.95 points in Year 1 and 0.89 points in Year 2. At Month 24, observed declines in both groups were numerically less than those predicted by natural-history models, although Capricor emphasized that these open-label analyses were descriptive and were not powered for formal Month 24 comparisons.
HOPE-3 Data Support Continued Deramiocel Development
Deramiocel is an investigational allogeneic cardiosphere-derived cell therapy designed to preserve skeletal and cardiac muscle function in DMD. The therapy consists of cardiac-derived cells that are intended to exert immunomodulatory and anti-fibrotic effects, including through secretion of extracellular vesicles known as exosomes. HOPE-3 is a Phase 3 randomized, double-blind, placebo-controlled study that enrolled 106 patients, with Deramiocel or placebo administered intravenously every three months during the 12-month randomized period. Patients completing the randomized portion were eligible to continue into the open-label extension, where all participants received Deramiocel.
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Deramiocel Advances Toward FDA Decision
The new HOPE-3 OLE findings have been included in a major amendment to Capricor’s Deramiocel Biologics License Application (BLA) submitted to the FDA, together with additional sensitivity and robustness analyses supporting the randomized clinical-trial results. Capricor is continuing discussions with the FDA as the regulatory review progresses toward a November 22, 2026 PDUFA target action date. Deramiocel has received Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations in the United States, as well as Orphan Drug and ATMP designations in Europe. The latest HOPE-3 data provide additional evidence supporting Deramiocel’s potential to slow upper limb functional decline in DMD as Capricor approaches the FDA’s regulatory decision.
Source:Capricor Therapeutics, press release
